PL1874306T3 - Kompozycje zawierające 1,2,4-oksadiazol aktywny po podaniu doustnie w celu supresyjnego leczenia niesensownej mutacji - Google Patents

Kompozycje zawierające 1,2,4-oksadiazol aktywny po podaniu doustnie w celu supresyjnego leczenia niesensownej mutacji

Info

Publication number
PL1874306T3
PL1874306T3 PL06749439T PL06749439T PL1874306T3 PL 1874306 T3 PL1874306 T3 PL 1874306T3 PL 06749439 T PL06749439 T PL 06749439T PL 06749439 T PL06749439 T PL 06749439T PL 1874306 T3 PL1874306 T3 PL 1874306T3
Authority
PL
Poland
Prior art keywords
oxadiazole
compositions
nonsense mutation
orally active
suppression therapy
Prior art date
Application number
PL06749439T
Other languages
English (en)
Polish (pl)
Inventor
Samit Hirawat
Langdon Miller
Original Assignee
Ptc Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ptc Therapeutics Inc filed Critical Ptc Therapeutics Inc
Publication of PL1874306T3 publication Critical patent/PL1874306T3/pl

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/4245Oxadiazoles
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/141Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/145Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic compounds
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    • A61P3/06Antihyperlipidemics
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    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
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    • A61P39/06Free radical scavengers or antioxidants
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    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • AHUMAN NECESSITIES
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    • A61P5/14Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
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    • A61P9/00Drugs for disorders of the cardiovascular system
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    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis

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  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
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  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Neurology (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
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  • Endocrinology (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Obesity (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Rheumatology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Oncology (AREA)
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  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Psychology (AREA)
  • Pain & Pain Management (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Child & Adolescent Psychology (AREA)
  • Ophthalmology & Optometry (AREA)
PL06749439T 2005-04-08 2006-04-06 Kompozycje zawierające 1,2,4-oksadiazol aktywny po podaniu doustnie w celu supresyjnego leczenia niesensownej mutacji PL1874306T3 (pl)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US66978905P 2005-04-08 2005-04-08
US78739506P 2006-03-30 2006-03-30
EP06749439A EP1874306B1 (en) 2005-04-08 2006-04-06 Compositions of an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy
PCT/US2006/012887 WO2006110483A1 (en) 2005-04-08 2006-04-06 Compositions of an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy

Publications (1)

Publication Number Publication Date
PL1874306T3 true PL1874306T3 (pl) 2012-11-30

Family

ID=36763092

Family Applications (2)

Application Number Title Priority Date Filing Date
PL06749439T PL1874306T3 (pl) 2005-04-08 2006-04-06 Kompozycje zawierające 1,2,4-oksadiazol aktywny po podaniu doustnie w celu supresyjnego leczenia niesensownej mutacji
PL11175569T PL2402002T3 (pl) 2005-04-08 2006-04-06 Kompozycje zawierające kwas 1,2,4-oksadiazolobenzoesowy i ich zastosowania do leczenia chorób związanych z przedwczesnym kodonem stop

Family Applications After (1)

Application Number Title Priority Date Filing Date
PL11175569T PL2402002T3 (pl) 2005-04-08 2006-04-06 Kompozycje zawierające kwas 1,2,4-oksadiazolobenzoesowy i ich zastosowania do leczenia chorób związanych z przedwczesnym kodonem stop

Country Status (16)

Country Link
US (7) US8716321B2 (pt)
EP (2) EP1874306B1 (pt)
JP (3) JP5800451B2 (pt)
CN (1) CN103720688A (pt)
AU (1) AU2006235115C1 (pt)
BR (1) BRPI0609089A2 (pt)
CA (1) CA2603402C (pt)
DK (2) DK2402002T3 (pt)
ES (2) ES2683359T3 (pt)
IL (2) IL186433A (pt)
MX (1) MX2007012206A (pt)
NO (2) NO339909B1 (pt)
NZ (1) NZ562197A (pt)
PL (2) PL1874306T3 (pt)
PT (2) PT2402002T (pt)
WO (1) WO2006110483A1 (pt)

Families Citing this family (51)

* Cited by examiner, † Cited by third party
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HUE047569T2 (hu) 2003-04-11 2020-04-28 Ptc Therapeutics Inc 1,2,4-oxadiazol-benzoesav vegyület és nonszensz szupresszióra történõ alkalmazása
PT2402002T (pt) 2005-04-08 2018-10-08 Ptc Therapeutics Inc Composições de 1,2,4-oxadiazole oralmente activo para terapia de supressão de mutações nonsense
RU2462246C2 (ru) 2006-03-30 2012-09-27 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы получения функционального белка из днк, имеющей нонсенс-мутацию, и лечения нарушений, ассоциированных с ней
NZ571803A (en) 2006-04-07 2011-12-22 Vertex Pharma Amide indole derivatives as modulators of ATP-binding cassette transporters
USRE50453E1 (en) 2006-04-07 2025-06-10 Vertex Pharmaceuticals Incorporated Indole derivatives as CFTR modulators
US10022352B2 (en) 2006-04-07 2018-07-17 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
US7645789B2 (en) 2006-04-07 2010-01-12 Vertex Pharmaceuticals Incorporated Indole derivatives as CFTR modulators
MX2009002439A (es) 2006-09-08 2009-03-27 Ptc Therapeutics Inc Procesos para la preparacion de acidos 1,2,4-oxadiazolbenzoicos.
US8101641B2 (en) 2006-09-25 2012-01-24 Ptc Therapeutics, Inc. Hydroxylated 1,2,4-oxadiazole benzoic acid compounds and compositions thereof
PL2076501T3 (pl) * 2006-09-25 2016-06-30 Ptc Therapeutics Inc Postacie krystaliczne kwasu 3-[5-(2-fluorofenylo)[1,2,4]oksadiazol-3-ilo]-benzoesowego
AU2016262645B2 (en) * 2006-10-12 2018-05-17 Ptc Therapeutics, Inc. Methods for dosing an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy
RS54025B1 (sr) * 2006-10-12 2015-10-30 Ptc Therapeutics Inc. Postupci doziranja oralno aktivnog 1,2,4-oksadiazola za supresionu terapiju nonsens mutacije
US8563573B2 (en) 2007-11-02 2013-10-22 Vertex Pharmaceuticals Incorporated Azaindole derivatives as CFTR modulators
GB0715087D0 (en) 2007-08-03 2007-09-12 Summit Corp Plc Drug combinations for the treatment of duchenne muscular dystrophy
CA2685540C (en) 2007-08-03 2018-10-16 Graham Michael Wynne Drug combinations for the treatment of duchenne muscular dystrophy
SI2217572T1 (sl) 2007-11-16 2014-05-30 Vertex Pharmaceuticals Incorporated Izokinolinski modulatorji prenaĺ alcev z atp-vezavno kaseto
SI2225230T1 (sl) 2007-12-07 2017-03-31 Vertex Pharmaceuticals Incorporated Trdne oblike 3-(6-(1-2,2-difluorobenzo(d)(1,3)dioxol-5-il)ciklopropan- karboksamido)-3-metilpiridin-2-il) benzojske kisline
EP2271621B1 (en) 2008-03-31 2013-11-20 Vertex Pharmaceuticals Incorporated Pyridyl derivatives as cftr modulators
US20110251253A1 (en) 2010-03-25 2011-10-13 Vertex Pharmaceuticals Incorporated Solid forms of (r)-1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)-n-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1h-indol-5-yl) cyclopropanecarboxamide
US8802868B2 (en) 2010-03-25 2014-08-12 Vertex Pharmaceuticals Incorporated Solid forms of (R)-1(2,2-difluorobenzo[D][1,3]dioxo1-5-yl)-N-(1-(2,3-dihydroxypropyl-6-fluoro-2-(1-hydroxy-2-methylpropan2-yl)-1H-Indol-5-yl)-Cyclopropanecarboxamide
HRP20211752T1 (hr) 2010-04-07 2022-02-18 Vertex Pharmaceuticals Incorporated Farmaceutski pripravci 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioksol-5-il)ciklopropankarboksamido)-3-metilpiridin-2-il)benzojeve kiseline i njihova primjena
HRP20160682T1 (hr) 2010-04-07 2016-07-29 Vertex Pharmaceuticals Incorporated Čvrsti oblici 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioksol-5-il)ciklopropankarboksiamido)-3-metilpiridin-2-il)benzojeve kiseline
ES2608474T3 (es) 2010-04-22 2017-04-11 Vertex Pharmaceuticals Incorporated Proceso de producción de compuestos indol cycloalkylcarboxamido
US8563593B2 (en) 2010-06-08 2013-10-22 Vertex Pharmaceuticals Incorporated Formulations of (R)-1-(2,2-difluorobenzo[D] [1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl)cyclopropanecarboxamide
JP5877200B2 (ja) 2010-08-05 2016-03-02 ユニヴェルシテ ドゥ ドロワ エ サンテ ドゥ リール ナンセンス突然変異による疾患の治療に有用な化合物及びその化合物を含む医薬組成物
CA2808501A1 (en) 2010-08-23 2012-03-01 Vertex Pharmaceuticals Incorporated Pharmaceutical composition of (r)-1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)-n-(1-(2,3-dihydroxy propyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1h-indol-5-yl) cyclopropanecarboxamide and administration therof
IN2014KN00885A (pt) 2011-11-08 2015-10-02 Vertex Pharma
WO2013112804A1 (en) 2012-01-25 2013-08-01 Vertex Pharmaceuticals Incorporated Formulations of 3-(6-(1-(2.2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid
WO2014014841A1 (en) 2012-07-16 2014-01-23 Vertex Pharmaceuticals Incorporated Pharmaceutical compositions of (r)-1-(2,2-diflurorbenzo[d][1,3]dioxol-5-yl)-n-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1h-indol-5-yl) cyclopropanecarboxamide and administration thereof
LT3470063T (lt) 2012-11-02 2025-02-10 Vertex Pharmaceuticals Incorporated Farmacinės kompozicijos, skirtos cftr nulemtų ligų gydymui
WO2014153643A1 (en) * 2013-03-26 2014-10-02 The University Of British Columbia Compositions and methods for use thereof in the treatment of aniridia
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